Dr. Shengdar Q. Tsai is an assistant member in the Department of Hematology at St. Jude Children’s Research Hospital. He obtained his B.S. in Biochemistry and Chemistry from the University of Michigan, M.S. in Bioinformatics and Ph.D. in Functional Genomics from North Carolina State University. He was a postdoctoral research fellow and instructor at Massachusetts General Hospital and Harvard Medical School. Dr. Tsai has focused on developing methods for high throughput genome editing with TALENs, and defining and improving the genome-wide specificity of CRISPR-Cas nucleases. His long-term research goals are to develop safe and highly specific targeted genome-editing strategies for treating human genetic diseases. His research interests include: • Improving genomic methods to define and measure gene editing “off-target” effects • Protein engineering of CRISPR-Cas nucleases for precise gene correction • Regulation of DNA damage repair choice between error-prone non-homologous end-joining and precise homology-directed repair • Genome-scale CRISPR-Cas screening for novel effectors of hematological diseases • Developing novel strategies for allele-specific editing of genetic mutations that cause sickle cell disease and other hematological disorders Online profile

Dr. Eric E. Bouhassira joined the Albert Einstein College of Medicine in 1990. He began studying human embryonic stem cells in 2001. He is Director of the Medical School's Center for Human Embryonic Stem Cell Research and Professor of Medicine and of Cell Biology. Dr. Bouhassira's research focuses on developing hematopoietic (blood forming) stem cells that can differentiate into red blood cells, T cells, platelets, and all other cell types that comprise blood. This work could potentially aid patients needing transfusions and also save lives by expanding the immunology diversity of hematopoietic stem cells available for transplant. Dr. Bouhassira is also interested in epigenetic regulation in the erythoid and hematopoietic lineage with a focus on DNA replication and DNA methylation. Dr. Bouhassira received his BS, MS, and PhD degrees from the Université Pierre et Marie Curie in Paris, France. Dr. Bouhassira also holds the Ingeborg and Ira Rennert Chair in Stem Cell Biology and Regenerative Medicine.   Areas of Research: Pluripotent stem cells, erythroid differentiation, red blood cells, epigenetics, DNA methylation, DNA replication, bioinformatics, allele-specific expression, gene therapy, hemoglobinopathies Online profile

These general caregiving tips provide families with information on how to stay healthy and positive. Keep in mind that these tips can be used to address many family issues. Information, support, advocacy, empowerment, care, and balance can be the foundation for a healthy family and are appropriate no matter what the challenge. Be Informed - Gather information about your family member’s condition, and discuss issues with others involved in the care of your family member. Being informed will help you make more knowledgeable health decisions and improve your understanding about any challenges your family might face. - Notice how others care for the person with special needs. Be aware of signs of mental or physical abuse. Get Support - Family members and friends can provide support in a variety of ways and oftentimes want to help. Determine if there are big or small things they can do to assist you and your family. - Join a local or online support group. A support group can give you the chance to share information and connect with people who are going through similar experiences. A support group may help combat the isolation and fear you may experience as a caregiver.   Abstract source

Maybe you take care of your spouse, who's in the early stages of Alzheimer's. Or you've been tending to the needs of your mother since she was diagnosed with cancer. Perhaps your child was born with a lifelong condition requiring specialized care. Whatever the circumstances, family caregivers tend to have at least one thing in common: They forget to take care of themselves. While caring for those you love is important, and can be meaningful, caregiving is stressful work. It can be exhausting, both physically and emotionally. If you don't take good care of yourself, you won't have the energy to care for others. Caregiver burnout Extended periods of providing care and support for a loved one can affect you in many ways, including: - Sleeplessness: Stress, anxiety or medical needs from your loved one in the middle of the night can make it difficult to get a good night's rest. - Reduced ability to concentrate: With so many tasks and responsibilities to manage you may find it difficult to concentrate. Brain fog makes every task more difficult. - Mood swings: You may have minor changes in mood. You feel angry one minute and then sad and helpless moments later. Irritability also is common.   Abstract source

Patrick M. Woster is professor of drug discovery and biomedical sciences at the South Carolina College of Pharmacy in Charleston, SC, and serves as the South Carolina SmartState Endowed Chair in Drug Discovery at the Medical University of South Carolina. He received a B.S. in pharmacy from the University of Nebraska Medical Center in 1978, and a Ph.D. in medicinal chemistry from the University of Nebraska - Lincoln in 1986. Professor Woster maintains an active research program that has been funded by several agencies, including the National Institutes of Health, the World Health Association and the pharmaceutical industry. He has been a member of ACS and the Division of Medicinal Chemistry since 1979. Woster's research interests include the design, synthesis and evaluation of antitumor agents as well as the development of analogues with potent activity against a variety of cancer cell lines. Most recently, he has been working to develop a new gene-modifying sickle cell disease treatment, which could be fast-tracked to human clinical trials in as little as three years. Online profile

Maria Hartleben is a licensed clinical social worker, currently working with Advocate’s Children Hospital, Oak Lawn, Illinois. She is trained to help patients and their parents to cope with illness and hospitalization through counseling, support groups, financial assistance and resource referral. Online profile

Julie Panepinto is the Director of the Division of Blood Diseases and Resources (DBDR) at the National Heart, Lung, and Blood Institute of the National Institutes of Health. Prior to joining the NHLBI in June 2021, as the Deputy Director of DBDR, Dr. Panepinto was a tenured Professor of Pediatrics, Hematology, Vice Chair of Value in Pediatrics, and the Director of the Center for Clinical Effectiveness Research of the Children’s Research Institute at the Medical College of Wisconsin and Children’s Wisconsin. As a clinician researcher with a background in public health, Dr. Panepinto focused her academic career on understanding and improving the health outcomes of patients and families with chronic diseases. Her research focused on integrating the perspective of the patient and family in health care through the use of patient reported outcomes (PROs) and understanding systems of care and acute care utilization for those individuals living with sickle cell disease. Dr. Panepinto completed her fellowship in Pediatric Hematology/Oncology/Bone Marrow Transplant at the University of Colorado Health Sciences in Denver, Colorado, where she also earned a Master of Science in Public Health. She served as a past member on the NHLBI Data and Safety Monitoring Board (DSMB) for the Sickle Cell Disease Clinical Research Network (2006-2010), the NHLBI’s Sickle Cell Disease Advisory Committee from 2016-2019, and the Advisory Council for the NHLBI from 2020-2021. Online profile

Dr. Gaurav Kharya is a Clinical Lead at Center for Bone Marrow Transplant and Cellular Therapy, Senior Consultant in pediatric hematology oncology and immunology at Indraprastha Apollo Hospitals, New Delhi. He completed his graduation from Netaji Subash Chandra Bose Medical College, Jabalpur Madhya Pradesh. Following this, he did his post-graduation DCH in pediatric from Baba Raghav Das Medical College, Gorakhpur, UP. In 2009, he joined his fellowship in pediatric hemato-oncology & BMT at Sir Ganga Ram Hospital which he completed in 2011. Dr. Kharya has a vast experience in transplanting children with various blood disorders, benign or malignant, immunological diseases etc nationally and internationally. Dr. Kharya is credited for doing the first haploidentical bone marrow transplant for sickle cell disease in India & also for doing in-vitro TCR alpha beta CD 19 depleted haploidentical BMT in a 4 month old baby suffering from severe combined immunodeficiency who is the smallest child transplanted in the country so far. Along with his team members at different centers, he has done more than 600 bone marrow transplants so far. His area of interest is alternative donor transplant such as haploidentical transplants and matched or mismatched unrelated donor transplants. Online profile

Dr. Dana LeBlanc specializes in pediatric hematology/oncology. She obtained her MD in 2009 from Louisiana State University School of Medicine. She completed her pediatrics residency in 2012 followed by her pediatric hematology/oncology fellowship in 2015, both from LSU Health Sciences Center/Children’s Hospital, New Orleans. Currently, Dr. LeBlanc is working as Assistant Professor of Clinical Pediatrics in the Department of Pediatrics at Children’s Hospital. Her clinical interests are sickle cell disease and non-malignant hematology. She also has a research interest in neurobiological mechanism of pain in sickle cell disease. Online profile

Donna is the Clinical Director at Arkansas Pregnancy Resource Center. Prior to this, she was the project manager at UAMS Adult Sickle Cell Clinical Program. She has been with Center for Distance Health/ANGELS since 2005, and served 5 years as the Manager of the ANGELS/CDH Triage Call Center that provides support for a campus-wide Physician Call Center, the ANGELS High Risk Ob Telemedicine Network and the innovative AR SAVES Telestroke program. Donna also facilitates the development of new innovative telehealth projects and trains healthcare practitioners across Arkansas how to use telemedicine equipment for clinical applications. Online profile